October 20, 2019

Positive Results From RGX-314 Gene Therapy

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Positive Results From RGX-314 Gene Therapy

(Updated 9/10/2020)

As reported here in 2017, a drug called RGX-314 is being tested as a one-time sub-retinal injection for wet age-related macular degeneration (wAMD). A preliminary study showed that patients with wAMD had long-term vision stabilization and anatomic improvement following a single treatment with the investigational gene therapy.

Now, in a subgroup of patients with the longest follow-up, three of six showed sustained benefit from a single injection of RGX-314 for more than 1.5 years. In another cohort with shorter follow-up, nine of 12 patients remained free of rescue medication for up to 6 months.

None of 42 patients treated in five dose cohorts had drug-related serious adverse events (SAEs). The results were reported at the American Academy of Ophthalmology meeting in October 2019.

Investigation of RGX-314 with a phase IIb trial (AAVIATE) is proceeding, with dosing of the first patient using suprachoroidal delivery. REGENXBIO expects to have an interim data update from the first cohort by the end of 2020. This targeted, in-office suprachoroidal delivery of RGX-314 using the SCS Microinjector® may provide additional administration options for patients with wet AMD.

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